Ignite Creation Date:
2025-12-24 @ 10:00 PM
Ignite Modification Date:
2025-12-29 @ 1:31 PM
Study NCT ID:
NCT00909532
Status:
COMPLETED
Last Update Posted:
2013-01-18
First Post:
2009-05-26
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}, {'id': 'D005355', 'term': 'Fibrosis'}, {'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D010335', 'term': 'Pathologic Processes'}], 'ancestors': [{'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C545203', 'term': 'ivacaftor'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medicalinfo@vrtx.com', 'phone': '617-444-6777', 'title': 'Medical Monitor', 'organization': 'Vertex'}, 'certainAgreement': {'restrictionType': 'GT60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'For enrolled subjects, adverse events were collected through the Follow-up Visit (4 weeks [± 7 days] after the last dose of study drug).', 'description': 'For subjects who were screened but were not subsequently enrolled in the study, non-serious AEs were not collected, but SAEs were reported. For subjects who completed 48 weeks of study drug treatment and enrolled in the open-label extension study, adverse events were only collected through the Week 48 Visit.', 'eventGroups': [{'id': 'EG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.', 'otherNumAtRisk': 78, 'otherNumAffected': 78, 'seriousNumAtRisk': 78, 'seriousNumAffected': 33}, {'id': 'EG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.', 'otherNumAtRisk': 83, 'otherNumAffected': 82, 'seriousNumAtRisk': 83, 'seriousNumAffected': 20}], 'otherEvents': [{'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 59, 'numAffected': 33}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 38, 'numAffected': 27}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 23, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 25, 'numAffected': 17}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 17, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 22, 'numAffected': 17}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 21, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 17, 'numAffected': 9}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 19, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 15, 'numAffected': 12}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Rales', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 14, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 17, 'numAffected': 9}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Respiratory tract congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 9, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Sinus congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 9, 'numAffected': 8}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Paranasal sinus hypersecretion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 7, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 8, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pleuritic pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Respiration abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 16, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 26, 'numAffected': 19}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 14, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 13, 'numAffected': 10}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 12, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 8, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 13, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 14, 'numAffected': 13}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 12, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 22, 'numAffected': 13}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 12, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 12, 'numAffected': 11}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 10, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 13, 'numAffected': 9}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 7, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pulmonary function test decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 14, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 6, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 9, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'C-reactive protein increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 6, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Blood glucose increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Bacteria sputum identified', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Breath sounds abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Cystic fibrosis lung', 'notes': 'CF exacerbations were coded as "cystic fibrosis lung."', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 77, 'numAffected': 39}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 47, 'numAffected': 29}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 31, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 39, 'numAffected': 19}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 11, 'numAffected': 10}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Sinus headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 11, 'numAffected': 7}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 7, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 22, 'numAffected': 12}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 11, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 13, 'numAffected': 10}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 9, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 8, 'numAffected': 7}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Joint sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Hypoglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}], 'seriousEvents': [{'term': 'Atrioventricular block complete', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Cystic fibrosis lung', 'notes': 'CF exacerbations were coded as "cystic fibrosis lung."', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 37, 'numAffected': 26}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 22, 'numAffected': 11}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pancreatic pseudocyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pancreatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Catheter related complication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Anaphylactic shock', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Implant site infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Lung infection pseudomonal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Myringitis bullous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Hepatic enzyme increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Hypoglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Cervix carcinoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Abortion spontaneous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'IgA nephropathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Renal colic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Testicular torsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Hemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pleuritic pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Pulmonary embolism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Respiratory distress', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 78, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '78', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.2', 'spread': '0.7', 'groupId': 'OG000'}, {'value': '10.4', 'spread': '0.7', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '10.6', 'ciLowerLimit': '8.6', 'ciUpperLimit': '12.6', 'pValueComment': "The primary and key secondary endpoints were analyzed using Hochberg's step-up procedure: test 1, primary (α=0.05); test 2, CFQ-R resp domain (Wk24) and sweat chloride (Wk24)(α=0.05).", 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.0', 'groupDescription': 'The primary analysis for the primary efficacy variable was based on a Mixed-Effects Model for Repeated Measures (MMRM). The model included absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) as the dependent variable, treatment (ivacaftor versus placebo) and visit (Day 15, Week 8, Week 16, and Week 24) as fixed effects, and subject as a random effect, with adjustment for the continuous baseline values of age and percent predicted FEV1.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': 'Denominator degrees of freedom were estimated using the Kenward-Roger approximation.', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline through 24 weeks', 'description': 'Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.', 'unitOfMeasure': 'percent of predicted volume (L)', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.'}, {'type': 'SECONDARY', 'title': 'Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '78', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablets of 150 mg of ivacaftor q12h for up to 48 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.4', 'spread': '0.7', 'groupId': 'OG000'}, {'value': '10.1', 'spread': '0.7', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '10.5', 'ciLowerLimit': '8.5', 'ciUpperLimit': '12.5', 'pValueComment': 'There was no adjustment for multiple comparisons.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.0', 'groupDescription': 'Analysis of this variable was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were obtained from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for the continuous baseline values of age and percent predicted forced expiratory volume in 1 second (FEV1),using unstructured covariance matrix.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': 'Denominator degrees of freedom were estimated using the Kenward-Roger approximation. No imputation of missing data was done.', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline through 48 weeks', 'description': 'Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.', 'unitOfMeasure': 'percent of predicted volume (L)', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame..'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '71', 'groupId': 'OG000'}, {'value': '80', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.'}], 'classes': [{'title': 'Change from Baseline Through Week 24', 'categories': [{'measurements': [{'value': '-2.1', 'spread': '1.3', 'groupId': 'OG000'}, {'value': '6.0', 'spread': '1.2', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline Through Week 48', 'categories': [{'measurements': [{'value': '-2.7', 'spread': '1.2', 'groupId': 'OG000'}, {'value': '6.0', 'spread': '1.1', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '8.1', 'ciLowerLimit': '4.7', 'ciUpperLimit': '11.4', 'pValueComment': "Analyzed in sequence: test 1, primary (α=0.05); test 2, using Hochberg's step-up procedure on CFQ-R resp domain(Wk 24) and sweat chloride (Wk 24) (α=0.05).", 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.7', 'groupDescription': 'Through Week 24: Analysis for the respiratory domain score endpoint was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for age, domain score, and percent predicted FEV1, using unstructured covariance matrix.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '8.6', 'ciLowerLimit': '5.3', 'ciUpperLimit': '11.9', 'pValueComment': 'There was no adjustment for multiple comparisons.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.7', 'groupDescription': 'Through Week 48: Analysis for the CFQ-R respiratory domain score endpoint was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from MMRM with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for age,sweat chloride, and percent predicted FEV1,using unstructured covariance matrix.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '74', 'groupId': 'OG000'}, {'value': '78', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.'}], 'classes': [{'title': 'Change from Baseline Through Week 24', 'categories': [{'measurements': [{'value': '-0.8', 'spread': '1.3', 'groupId': 'OG000'}, {'value': '-48.7', 'spread': '1.2', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline Through Week 48', 'categories': [{'measurements': [{'value': '-0.6', 'spread': '1.3', 'groupId': 'OG000'}, {'value': '-48.7', 'spread': '1.2', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-47.9', 'ciLowerLimit': '-51.3', 'ciUpperLimit': '-44.5', 'pValueComment': "Analyzed in sequence: test 1, primary (α=0.05); test 2, using Hochberg's step-up procedure on CFQ-R resp domain(Wk 24) and sweat chloride (Wk 24) (α=0.05); test 3 using Hochberg's on time to pulmonary exacerbation (Wk 48) and weight (Wk 48).", 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.7', 'groupDescription': 'Through Week 24: Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for age, sweat chloride, and percent predicted FEV1, using unstructured covariance matrix.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-48.1', 'ciLowerLimit': '-51.5', 'ciUpperLimit': '-44.7', 'pValueComment': 'There was no adjustment for multiple comparisons.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.7', 'groupDescription': 'Through Week 48: Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for age, sweat chloride, and percent predicted FEV1, using unstructured covariance matrix.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.', 'unitOfMeasure': 'millimoles per liter', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame..'}, {'type': 'SECONDARY', 'title': 'Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '78', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.'}], 'classes': [{'title': '0 to 15 Days', 'categories': [{'measurements': [{'value': '0.97', 'groupId': 'OG000', 'lowerLimit': '0.90', 'upperLimit': '0.99'}, {'value': '0.98', 'groupId': 'OG001', 'lowerLimit': '0.91', 'upperLimit': '0.99'}]}]}, {'title': '16 to 56 Days', 'categories': [{'measurements': [{'value': '0.87', 'groupId': 'OG000', 'lowerLimit': '0.77', 'upperLimit': '0.93'}, {'value': '0.89', 'groupId': 'OG001', 'lowerLimit': '0.80', 'upperLimit': '0.94'}]}]}, {'title': '57 to 112 Days', 'categories': [{'measurements': [{'value': '0.72', 'groupId': 'OG000', 'lowerLimit': '0.61', 'upperLimit': '0.81'}, {'value': '0.83', 'groupId': 'OG001', 'lowerLimit': '0.73', 'upperLimit': '0.90'}]}]}, {'title': '113 to 168 Days', 'categories': [{'measurements': [{'value': '0.53', 'groupId': 'OG000', 'lowerLimit': '0.41', 'upperLimit': '0.64'}, {'value': '0.78', 'groupId': 'OG001', 'lowerLimit': '0.68', 'upperLimit': '0.86'}]}]}, {'title': '169 to 224 Days', 'categories': [{'measurements': [{'value': '0.51', 'groupId': 'OG000', 'lowerLimit': '0.39', 'upperLimit': '0.61'}, {'value': '0.75', 'groupId': 'OG001', 'lowerLimit': '0.64', 'upperLimit': '0.83'}]}]}, {'title': '225 to 280 Days', 'categories': [{'measurements': [{'value': '0.44', 'groupId': 'OG000', 'lowerLimit': '0.32', 'upperLimit': '0.55'}, {'value': '0.70', 'groupId': 'OG001', 'lowerLimit': '0.58', 'upperLimit': '0.78'}]}]}, {'title': '281 to 336 Days', 'categories': [{'measurements': [{'value': '0.41', 'groupId': 'OG000', 'lowerLimit': '0.29', 'upperLimit': '0.52'}, {'value': '0.67', 'groupId': 'OG001', 'lowerLimit': '0.55', 'upperLimit': '0.76'}]}]}], 'analyses': [{'pValue': '0.0016', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Cox Proportional Hazard at Week 24', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.40', 'ciLowerLimit': '0.23', 'ciUpperLimit': '0.71', 'pValueComment': 'There was no adjustment for multiple comparisons.', 'groupDescription': 'Time to first pulmonary exacerbation through Week 24 was analyzed using Cox regression. The model included a covariate for treatment and adjustments for the age group and percent predicted forced expiratory volume in 1 second (FEV1) severity at baseline.', 'statisticalMethod': 'Regression, Cox', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '0.0012', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Cox Proportional Hazard at 48 Weeks', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.46', 'ciLowerLimit': '0.28', 'ciUpperLimit': '0.73', 'pValueComment': "Analyzed in sequence: test 1, primary (α=0.05); test 2, using Hochberg's step-up procedure on CFQ-R resp domain(Wk 24) and sweat chloride (Wk 24) (α=0.05); test 3 using Hochberg's on time to pulmonary exacerbation (Wk 48) and weight (Wk 48).", 'groupDescription': 'Time to first pulmonary exacerbation through Week 48 was analyzed using Cox regression. The model included a covariate for treatment and adjustments for the age group and percent predicted forced expiratory volume in 1 second (FEV1) severity at baseline.', 'statisticalMethod': 'Regression, Cox', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'NUMBER', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection.', 'unitOfMeasure': 'proportion of event-free participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Weight at Week 24 and Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '78', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.'}], 'classes': [{'title': 'At Week 24', 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.4', 'groupId': 'OG000'}, {'value': '3.0', 'spread': '0.4', 'groupId': 'OG001'}]}]}, {'title': 'At Week 48', 'categories': [{'measurements': [{'value': '0.4', 'spread': '0.5', 'groupId': 'OG000'}, {'value': '3.1', 'spread': '0.5', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.8', 'ciLowerLimit': '1.8', 'ciUpperLimit': '3.7', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.5', 'groupDescription': 'At Week 24: Analysis for this variable was based on a linear mixed effects (LME) model with treatment as a fixed effect, and intercept, visit (days on study) and treatment by visit interaction as random effects, with adjustment for age group and baseline percent predicted forced expiratory volume in 1 second (FEV1) severity.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': 'There was no adjustment for multiple comparisons.', 'testedNonInferiority': False}, {'pValue': '0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.7', 'ciLowerLimit': '1.3', 'ciUpperLimit': '4.1', 'pValueComment': "Analyzed in sequence: test 1, primary (α=0.05); test 2, using Hochberg's step-up procedure on CFQ-R resp domain(Wk 24) and sweat chloride (Wk 24) (α=0.05).", 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.7', 'groupDescription': 'At Week 48: Analysis for this variable was based on a linear mixed effects (LME) model with treatment as a fixed effect and visit (days on study) and treatment by visit interaction as random effects, with adjustment for age group and baseline percent predicted forced expiratory volume in 1 second (FEV1) severity.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline to 24 weeks and 48 weeks', 'description': 'As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.', 'unitOfMeasure': 'kilograms', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.'}, {'id': 'FG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'All subjects who received at least 1 dose of study drug (placebo).', 'groupId': 'FG000', 'numSubjects': '78'}, {'comment': 'All subjects who received at least 1 dose of study drug (ivacaftor).', 'groupId': 'FG001', 'numSubjects': '83'}]}, {'type': 'Completed Treatment Period, Week 24', 'achievements': [{'groupId': 'FG000', 'numSubjects': '71'}, {'groupId': 'FG001', 'numSubjects': '80'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Completed Treatment and Extension Periods (Through Week 48)', 'groupId': 'FG000', 'numSubjects': '68'}, {'comment': 'Completed Treatment and Extension Periods (Through Week 48)', 'groupId': 'FG001', 'numSubjects': '77'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}, {'groupId': 'FG001', 'numSubjects': '6'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Pregnancy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Prohibited Medication', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal of Consent', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Noncompliance with Study Requirements', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Wrong Genotype', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Increased Lab Draws, Difficult Lab Stick', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'The study started on 10 June 2009 (signing of first informed consent). After obtaining consent and assent (where applicable), screening evaluations were completed during a period of 2 to 5 weeks (Day -35 to Day -15) before the first dose of study drug.', 'preAssignmentDetails': 'A total of 167 subjects were randomized; 161 subjects received at least 1 dose of the study drug. A 2-week run-in period was included to establish the baseline assessments on Day 1 after ensuring that subjects were properly taking their cystic fibrosis (CF) medication regimens.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '78', 'groupId': 'BG000'}, {'value': '83', 'groupId': 'BG001'}, {'value': '161', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks.'}, {'id': 'BG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '19', 'groupId': 'BG001'}, {'value': '36', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '61', 'groupId': 'BG000'}, {'value': '64', 'groupId': 'BG001'}, {'value': '125', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age Continuous', 'classes': [{'categories': [{'measurements': [{'value': '24.7', 'spread': '9.21', 'groupId': 'BG000'}, {'value': '26.2', 'spread': '9.85', 'groupId': 'BG001'}, {'value': '25.5', 'spread': '9.54', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '40', 'groupId': 'BG000'}, {'value': '44', 'groupId': 'BG001'}, {'value': '84', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '38', 'groupId': 'BG000'}, {'value': '39', 'groupId': 'BG001'}, {'value': '77', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '77', 'groupId': 'BG000'}, {'value': '81', 'groupId': 'BG001'}, {'value': '158', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '77', 'groupId': 'BG000'}, {'value': '81', 'groupId': 'BG001'}, {'value': '158', 'groupId': 'BG002'}]}]}, {'title': 'Not Allowed to Ask Per Local Regulations', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'North America', 'categories': [{'measurements': [{'value': '50', 'groupId': 'BG000'}, {'value': '50', 'groupId': 'BG001'}, {'value': '100', 'groupId': 'BG002'}]}]}, {'title': 'Europe', 'categories': [{'measurements': [{'value': '19', 'groupId': 'BG000'}, {'value': '23', 'groupId': 'BG001'}, {'value': '42', 'groupId': 'BG002'}]}]}, {'title': 'Australia', 'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '19', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Percent Predicted Forced Expiratory Volume in 1 Second (FEV1), Continuous', 'classes': [{'categories': [{'measurements': [{'value': '63.7', 'spread': '16.83', 'groupId': 'BG000'}, {'value': '63.5', 'spread': '16.14', 'groupId': 'BG001'}, {'value': '63.6', 'spread': '16.43', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'description': 'Percent predicted for age, gender, and height.', 'unitOfMeasure': 'percentage', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Percent Predicted FEV1, Categorical', 'classes': [{'title': '< 70% predicted FEV1', 'categories': [{'measurements': [{'value': '45', 'groupId': 'BG000'}, {'value': '49', 'groupId': 'BG001'}, {'value': '94', 'groupId': 'BG002'}]}]}, {'title': '≥ 70% predicted FEV1', 'categories': [{'measurements': [{'value': '33', 'groupId': 'BG000'}, {'value': '34', 'groupId': 'BG001'}, {'value': '67', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'description': 'Percent predicted for age, gender, and height.', 'unitOfMeasure': 'participants'}, {'title': 'Weight', 'classes': [{'categories': [{'measurements': [{'value': '61.2', 'spread': '13.93', 'groupId': 'BG000'}, {'value': '61.7', 'spread': '14.26', 'groupId': 'BG001'}, {'value': '61.5', 'spread': '14.06', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kilograms', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Body Mass Index', 'classes': [{'categories': [{'measurements': [{'value': '21.9', 'spread': '3.49', 'groupId': 'BG000'}, {'value': '21.7', 'spread': '3.65', 'groupId': 'BG001'}, {'value': '21.8', 'spread': '3.56', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kilograms per square meter', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sweat Chloride', 'classes': [{'categories': [{'measurements': [{'value': '100.1', 'spread': '10.63', 'groupId': 'BG000'}, {'value': '100.4', 'spread': '10.00', 'groupId': 'BG001'}, {'value': '100.2', 'spread': '10.28', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'millimoles per liter', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 167}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-01', 'dispFirstSubmitDate': '2011-09-21', 'completionDateStruct': {'date': '2012-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2013-01-14', 'studyFirstSubmitDate': '2009-05-26', 'dispFirstSubmitQcDate': '2011-09-21', 'resultsFirstSubmitDate': '2012-02-27', 'studyFirstSubmitQcDate': '2009-05-26', 'dispFirstPostDateStruct': {'date': '2011-09-26', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2013-01-18', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2012-07-18', 'studyFirstPostDateStruct': {'date': '2009-05-28', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2012-08-21', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24', 'timeFrame': 'baseline through 24 weeks', 'description': 'Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.'}], 'secondaryOutcomes': [{'measure': 'Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48', 'timeFrame': 'baseline through 48 weeks', 'description': 'Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.'}, {'measure': 'Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).'}, {'measure': 'Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.'}, {'measure': 'Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection.'}, {'measure': 'Absolute Change From Baseline in Weight at Week 24 and Week 48', 'timeFrame': 'baseline to 24 weeks and 48 weeks', 'description': 'As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Fibrosis', 'Pancreatic Diseases', 'Lung Diseases', 'Respiratory Tract Diseases', 'Genetic Diseases, Inborn', 'Infant, Newborn, Diseases', 'Pathologic Processes'], 'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '22047557', 'type': 'BACKGROUND', 'citation': 'Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.'}, {'pmid': '28651844', 'type': 'DERIVED', 'citation': 'Flume PA, Wainwright CE, Elizabeth Tullis D, Rodriguez S, Niknian M, Higgins M, Davies JC, Wagener JS. Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor. J Cyst Fibros. 2018 Jan;17(1):83-88. doi: 10.1016/j.jcf.2017.06.002. Epub 2017 Jun 24.'}, {'pmid': '27097977', 'type': 'DERIVED', 'citation': 'Solem CT, Vera-Llonch M, Liu S, Botteman M, Castiglione B. Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis. Health Qual Life Outcomes. 2016 Apr 21;14:63. doi: 10.1186/s12955-016-0465-z.'}, {'pmid': '26135562', 'type': 'DERIVED', 'citation': 'Quittner A, Suthoff E, Rendas-Baum R, Bayliss MS, Sermet-Gaudelus I, Castiglione B, Vera-Llonch M. Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial. Health Qual Life Outcomes. 2015 Jul 2;13:93. doi: 10.1186/s12955-015-0293-6.'}], 'seeAlsoLinks': [{'url': 'http://ghr.nlm.nih.gov/', 'label': 'Genetics Home Reference'}, {'url': 'http://www.nlm.nih.gov/medlineplus/', 'label': 'Medline Plus'}, {'url': 'http://www.clinicaltrials.gov/ct2/info/fdalinks', 'label': 'U.S. FDA Resources'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.', 'detailedDescription': 'This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%.\n\nBased on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.\n\nThis study was designed to further evaluate the efficacy of ivacaftor in subjects with CF who have a G551D-CFTR gene mutation and to evaluate safety in this population over a longer period than previously studied.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele\n* Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.\n* No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator\n* Willing to use highly effective birth control methods during the study\n\nExclusion Criteria:\n\n* History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject\n* Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study\n* History of alcohol, medication or illicit drug abuse within one year prior to Day 1\n* Abnormal liver function ≥ 3x the upper limit of normal\n* Abnormal renal function at Screening\n* History of solid organ or hematological transplantation\n* Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements\n* Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening\n* Use of inhaled hypertonic saline treatment\n* Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)'}, 'identificationModule': {'nctId': 'NCT00909532', 'acronym': 'STRIVE', 'briefTitle': 'Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation', 'orgStudyIdInfo': {'id': 'VX08-770-102'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Subjects who received placebo every 12 hours (q12h) for up to 48 weeks.', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': '150 mg Ivacaftor q12h', 'description': 'Subjects who received 150 mg of ivacaftor q12h for up to 48 weeks.', 'interventionNames': ['Drug: Ivacaftor']}], 'interventions': [{'name': 'Ivacaftor', 'type': 'DRUG', 'otherNames': ['VX-770'], 'description': '150-mg tablets given orally q12h for up to 48 weeks', 'armGroupLabels': ['150 mg Ivacaftor q12h']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Tablet given orally q12h for up to 48 weeks', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233-1711', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '94611', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': 'Kaiser Permanente Medical Care Program', 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Cystic Fibrosis Research Office, Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '92123-5070', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': 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